One-Time treatment aims to halt devastating childhood disease
NCT ID NCT03488394
Summary
This study is testing a one-time gene therapy for children with Hurler syndrome, a severe and life-limiting genetic disorder. Doctors collect a child's own blood stem cells, add a corrected gene in the lab, and then return the cells to the child. The main goals are to see if this treatment is safe and if it can provide the missing enzyme needed to control the disease for years.
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Contacts and locations
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Locations
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Ospedale San Raffaele
Milan, 20132, Italy
Conditions
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